Laboratorio de Patogénesis Molecular, Laboratorio 4, Edificio A4, Carrera Médico Cirujano, Facultad de Estudios Superiores Iztacala, Universidad Nacional Autónoma de México, Tlalnepantla 54090, Mexico.
Red MEDICI, Carrera Médico Cirujano, Facultad de Estudios Superiores Iztacala, Universidad Nacional Autónoma de México, Tlalnepantla 54090, Mexico.
Int J Mol Sci. 2023 Feb 7;24(4):3264. doi: 10.3390/ijms24043264.
Parkinson's disease (PD) is the most common α-synucleinopathy worldwide. The pathognomonic hallmark of PD is the misfolding and propagation of the α-synuclein (α-syn) protein, observed in post-mortem histopathology. It has been hypothesized that α-synucleinopathy triggers oxidative stress, mitochondrial dysfunction, neuroinflammation, and synaptic dysfunction, leading to neurodegeneration. To this date, there are no disease-modifying drugs that generate neuroprotection against these neuropathological events and especially against α-synucleinopathy. Growing evidence suggests that peroxisome proliferator-activated receptor (PPAR) agonists confer neuroprotective effects in PD, however, whether they also confer an anti-α-synucleinopathy effect is unknown. Here we analyze the reported therapeutic effects of PPARs, specifically the gamma isoform (PPARγ), in preclinical PD animal models and clinical trials for PD, and we suggest possible anti-α-synucleinopathy mechanisms acting downstream from these receptors. Elucidating the neuroprotective mechanisms of PPARs through preclinical models that mimic PD as closely as possible will facilitate the execution of better clinical trials for disease-modifying drugs in PD.
帕金森病(PD)是全球最常见的α-突触核蛋白病。PD 的特征性标志是α-突触核蛋白(α-syn)蛋白的错误折叠和传播,这在死后组织病理学中观察到。有人假设α-突触核蛋白病会引发氧化应激、线粒体功能障碍、神经炎症和突触功能障碍,导致神经退行性变。迄今为止,还没有能够针对这些神经病理事件,特别是针对α-突触核蛋白病产生神经保护作用的疾病修饰药物。越来越多的证据表明,过氧化物酶体增殖物激活受体(PPAR)激动剂在 PD 中具有神经保护作用,但是否也具有抗α-突触核蛋白病作用尚不清楚。在这里,我们分析了 PPARs(特别是γ亚型(PPARγ))在 PD 动物模型和临床试验中的报告治疗效果,并提出了可能的下游抗α-突触核蛋白病机制。通过尽可能模拟 PD 的临床前模型阐明 PPARs 的神经保护机制,将有助于更好地执行 PD 疾病修饰药物的临床试验。